FDA Advisers Consider New Lupus Drug

TUESDAY Nov. 16, 2010 -- U.S. Food and Drug Administration advisers were weighing Tuesday whether to recommend the first new drug in more than 50 years to treat the autoimmune disease lupus.

The drug, belimumab (Benlysta), is a so-called monoclonal antibody drug developed to treat patients who suffer from systemic lupus erythematosus.

In research papers submitted to the advisory committee, the full FDA expressed some concerns about the efficacy and safety of the drug.

"I really do think the drug is efficacious," said Sandra C. Raymond, president & CEO of the Lupus Foundation of America, who was attending the advisory panel meeting. "What I'm hearing in terms of efficacy is pretty strong."

The FDA said it wants the advisory panel to look at both the efficacy data, which according to the agency is modest, and the safety data, which the agency believes raises some serious issues. Specifically, the drug has been linked to depression and suicide.

Raymond noted that many patients with lupus suffer from depression. And many patients in clinical trials testing the drug were also taking antidepressants, she added.

The advisory committee is scheduled to vote on whether the drug's risks are mitigated by the benefits it has to offer to patients who are already receiving standard care.

The agency said it is basing its concerns on the results of two phase 3 trials that compared Benlysta with a placebo.

"The risks associated with belimumab include infection, malignancy, suicidality, and overall number of deaths with a numerical imbalance that favored placebo treatment over belimumab treatment," according to the FDA.

Safety is important, Raymond said, but the drugs used to treat lupus now carry some significant side effects, too. "The safety of some of our medications is not good," she said. "So what are we comparing it to?"

The current treatments include chemotherapy and steroids, which can leave the door open to infections, cancers and other unwanted consequences, Raymond said.

Dr. Joan Merrill, head of Clinical Pharmacology at the Oklahoma Medical Research Foundation and medical director of the Lupus Foundation of America, added that "the safety data on Benlysta looks better than we usually see with biologics."

"We have to keep in mind that there is a natural ceiling on the percentage of patients who can respond to any targeted treatment. We do not yet know exactly how good Benlysta is," Merrill said.

What is known is that Benlysta was shown to be statistically superior to a placebo in two phase 3 trials, she said.

"I hope that the FDA will be able to approve it, after they complete the serious deliberations that they must make on all the data," Merrill said.

Lupus is an autoimmune disease in which the body produces antibodies that attack and destroy healthy tissue. This can damage joints, skin, blood vessels and organs. Benlysta works by inhibiting the production of these antibodies.

Benlysta is made by Human Genome Sciences and GlaxoSmithKline.

While the FDA is not bound by its advisory committees' recommendations it usually does so.